Skip to main content
PortfolioPoseida Therapeutics, Inc.

Poseida Therapeutics: FDA Grants Poseida’s P-BCMA-101 Regenerative Medicine Advanced Therapy (RMAT) Status for Treatment of Multiple Myeloma

By November 19, 2018March 25th, 2021No Comments

The U.S. Food and Drug Administration (FDA) has granted P-BCMA-101Poseida Therapeutics’ CAR T-cell therapy and lead product candidate, the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of relapsed or refractory multiple myeloma.

Preliminary results from a Phase 1 trial (NCT03288493) suggest the candidate is safe and may have superior effectiveness compared with other CAR T-cell therapies.

The RMAT designation is granted to regenerative therapies intended to treat, modify, reverse, or cure a serious condition, and for which preliminary clinical data evidences the therapy’s potential to address unmet medical needs for a condition.

The statute grants all the benefits of a Fast Track and Breakthrough Therapy designation, including early interactions with sponsors and the FDA, which may be used to discuss intermediate or surrogate clinical trial endpoints and support accelerated approval of the therapy.

P-BCMA-101 is designed to target the B cell maturation antigen (BCMA), a protein highly expressed at the surface of virtually all multiple myeloma cells.

The therapy is based on the patient’s own T-cells, which are taken from the blood and genetically engineered to recognize BCMA and destroy cells producing this factor.

The patient’s T-cells are genetically modified through a molecular system owned by Poseida called piggyBac. This approach has the benefit of being virus-free and allowing the production of a highly pure population of stem cell memory T cells — proliferative cells capable of sustaining the therapy’s effect after its administration.

The high purity of P-BCMA-101 may reduce its toxicity compared with other CAR-T approaches, Poseida states. Also, P-BCMA-101 contains a “switch” so that the therapy can be rapidly reduced or eliminated in case of dangerous side effects.

The safety, dose tolerability and cancer response to the therapy is being tested in a Phase 1 clinical trial (NCT03288493) in patients with multiple myeloma whose cancer has returned after treatment (relapsed) or failed to respond to prior therapies (refractory).

Preliminary results from the study suggest that P-BCMA-101 may lead to superior response rates compared to other CAR-T therapies at similar doses, while presenting a positive safety profile.

“P-BCMA-101 is the first anti-BCMA CAR-T therapy to receive RMAT designation from the FDA and underscores the urgent need for new treatment options for multiple myeloma,” Eric Ostertag, MD, PhD, chief executive officer of Poseida Therapeutics, said in a press release.

He added that “initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population, with some patients reaching VGPR [very good partial response] and stringent CR [complete response].”

Updated results on the trial are expected by the end of 2018, said Ostertag, who added that Poseida is looking forward to working with the FDA to speed development of P-BCMA-101.

Rapha Capital Management, LLC is providing this website for informational purposes only. Nothing on this website is intended to constitute, or should be deemed to be, financial advisory, legal, tax, accounting or other professional advice to you or any other party; be an opinion of the appropriateness or suitability of any investment; or constitute an offer to sell or solicitation of an offer to buy any security or investment. This website and its content (collectively, “Content”) is intended for general informational purposes only and should not be acted upon without first obtaining financial advisory, legal, tax, accounting or other professional advice from your consultant or other professional with respect to your own personal investment objectives, financial circumstances and needs. Nothing on the Site shall be considered a recommendation or solicitation to buy or an offer to sell a security to any person in any jurisdiction where such offer, solicitation, purchase or sale would be unlawful under the securities laws of such jurisdiction. Any investment decisions you may make on the basis of any information found on this web site is your sole responsibility. Prior to making any investment or hiring any investment manager you should consult with a professional financial advisor, legal and tax advisor to assist in due diligence as may be appropriate and determining the appropriateness of the risk associated with a particular investment. In no event shall Rapha Capital Management, LLC be responsible or liable for the correctness of any such material or for any damage or lost opportunities resulting from use of these data.